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Updated: Jan 13, 2026

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向凝血因子XI的RNA干扰疗法 - - 这是RBD405959的第一次在人体上的试验

Anders Gabrielsen¹, Sebastian Ueckert¹, Catarina Nilsson¹

¹Ribocure Pharmaceuticals AB, Gothenburg, Sweden.

|January 9, 2026

概括

一种针对XI因子 (FXI) 的新型RNA干扰疗法,RBD4059,在首次人体试验中显示出卓越的安全性,并实现显著的,持久的FXI活性降低. 这支持其在预防血栓形成方面的潜力.

科学领域:

药理学药理学是指药理学的学科.
血栓形成研究研究
抗RNA干扰治疗药物抗RNA干扰治疗

背景情况:

血管内血栓形成是一个重大的临床挑战.
抑制凝血因子XI (FXI) 是一个有前途的抗血栓策略.
RNA干扰 (RNAi) 提供了一个有针对性的治疗方法.

研究的目的:

为了评估RBD4059首次在人体中使用的安全性和耐受性,RBD4059是一种针对FXI的N-乙糖氨酸 (GalNAc) 结合的小干扰RNA (siRNA).
在健康志愿者中评估RBD4059单次上升剂量的药理动力学和药理动力学影响.

主要方法:

第1期,单次上升剂量,随机化,安慰剂对照研究 (NCT05653037).
健康的志愿者接受皮下剂量RBD4059 (50毫克至600毫克) 或安慰剂.
安全性,耐受性,药理动力学和药理动力学参数被监测到169天后剂量.

主要成果:

单次上升剂量的RBD4059耐受性良好,没有发现安全问题.
RBD4059证明了剂量依赖且持久的FXI活性抑制,超过90%.
药理动力学建模建议3-6个月的剂量间隔持续抗血栓作用.

结论:

RBD4059是安全的,并且有效地抑制了FXI活动.
这些发现支持RBD4059的进一步临床开发,作为用于预防血栓形成的基于siRNA的疗法.
RBD4059可能为患有血栓形成风险的患者提供一种新的治疗选择.

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